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Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis

This study has been completed.
SciClone Pharmaceuticals
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: January 2000

OBJECTIVES: I. Evaluate the safety of ascending doses of CPX administered to adult patients with mild cystic fibrosis.

II. Evaluate the pharmacokinetics of ascending doses of CPX in this patient population.

Condition Intervention Phase
Cystic Fibrosis
Drug: CPX
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 35
Study Start Date: September 1997
Estimated Study Completion Date: October 1999
Detailed Description:

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, dose escalation study.

There are 7 experimental cohorts, each treated with a different oral dose of CPX or placebo. Within each cohort, 4 patients receive a single dose of CPX and 1 patient receives placebo. Each patient is monitored 24 hours postdose. Escalation to the next dose level for each subsequent cohort begins only after the safety data obtained from the previous cohort is reviewed and found not to limit dose escalation.

All patients return for a follow up evaluation 1 week after dosing.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Mild cystic fibrosis
  • Not pregnant or nursing Negative pregnancy test
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004428

Sponsors and Collaborators
SciClone Pharmaceuticals
Study Chair: Eduardo Martins SciClone Pharmaceuticals
  More Information

No publications provided Identifier: NCT00004428     History of Changes
Other Study ID Numbers: 199/13364, SCICLONE-FDA-OP-97-1
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Cystic Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases processed this record on November 27, 2014